EIPA

Spinal muscular atrophy (SMA), a disease in which muscle-controlling nerve cells (motor neurons) in the spinal cord are lost, is caused by a lack of full-length SMN, a protein normally produced from DNA instructions in the SMN1 gene. People with SMA lack SMN1 genes but have SMN2 genes, from which the majority of SMN protein molecules produced are relatively short compared to full-length SMN and...